Back to resultsZX008 Expanded Access Protocol
Primary Purpose
Dravet Syndrome
Status
Approved for marketing
Phase
Locations
Study Type
Expanded Access
Intervention
Fenfluramine Hydrochloride
Sponsored by
About this trial
This is an expanded access trial for Dravet Syndrome
Eligibility Criteria
Inclusion Criteria:
- Patient is male or female, age 2 years and older, inclusive as of Study Day 1.
- Patient is diagnosed with Dravet syndrome.
- Patient is experiencing convulsive seizures which are not controlled by current AEDs.
- Patient is receiving at least one AED and will remain on at least one AED for the duration of treatment.
- Patient has been approved for inclusion by Zogenix.
Exclusion Criteria:
- Patient requires or starts using an unacceptable or contraindicated concomitant medication.
- Patient has valvulopathy.
- Patient is at risk for pulmonary hypertension.
- Patient exclusion will be at the sole discretion of the Sponsor.
Sites / Locations
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT03780127
Brief Title
ZX008 Expanded Access Protocol
Official Title
ZX008 Expanded Access Protocol - Dravet Syndrome Treatment Plan
Study Type
Expanded Access
2. Study Status
Record Verification Date
October 2023
Overall Recruitment Status
Approved for marketing
Study Start Date
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Primary Completion Date
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Study Completion Date
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3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Zogenix, Inc.
4. Oversight
5. Study Description
Brief Summary
The treatment plan for this Expanded Access Protocol is for patients with Dravet syndrome who do not qualify for participation in one of the ongoing ZX008 clinical trials.
Detailed Description
The treatment plan consists of an up to 24-month Treatment Period. Access is via application by your health care provider and available at one of the Expanded Access treatment centers. The dose of ZX008 for the duration of the Treatment Period will range from 0.2 mg/kg/day to a maximum of 0.8 mg/kg/day, not to exceed a total daily dose of 30 mg/day; for patients concurrently being prescribed stiripentol, the maximum will be 0.5mg/kg/day, not to exceed a total daily dose of 20 mg/day.
The fenfluramine US Expanded Access Program (US EAP) is available to full-time US Residents only.
Participation in the US EAP is limited to patients currently residing in the US. Residency in the US must be for the duration of participation in the US EAP. Drug may not be shipped outside the US.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Dravet Syndrome
7. Study Design
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
Fenfluramine Hydrochloride
Other Intervention Name(s)
ZX008
Intervention Description
ZX008 drug product is an oral aqueous solution of fenfluramine hydrochloride. The product is sugar free and is intended to be compatible with KD.
10. Eligibility
Sex
All
Minimum Age & Unit of Time
2 Years
Eligibility Criteria
Inclusion Criteria:
Patient is male or female, age 2 years and older, inclusive as of Study Day 1.
Patient is diagnosed with Dravet syndrome.
Patient is experiencing convulsive seizures which are not controlled by current AEDs.
Patient is receiving at least one AED and will remain on at least one AED for the duration of treatment.
Patient has been approved for inclusion by Zogenix.
Exclusion Criteria:
Patient requires or starts using an unacceptable or contraindicated concomitant medication.
Patient has valvulopathy.
Patient is at risk for pulmonary hypertension.
Patient exclusion will be at the sole discretion of the Sponsor.
12. IPD Sharing Statement
Citations:
PubMed Identifier
34768178
Citation
Cross JH, Galer BS, Gil-Nagel A, Devinsky O, Ceulemans B, Lagae L, Schoonjans AS, Donner E, Wirrell E, Kothare S, Agarwal A, Lock M, Gammaitoni AR. Impact of fenfluramine on the expected SUDEP mortality rates in patients with Dravet syndrome. Seizure. 2021 Dec;93:154-159. doi: 10.1016/j.seizure.2021.10.024. Epub 2021 Nov 2.
Results Reference
derived
Learn more about this trial
ZX008 Expanded Access Protocol
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